ATE437944T1 - DNA SEQUENCES CODING FOR DYSTROPHIN MINIGEN AND METHOD FOR USE THEREOF - Google Patents

DNA SEQUENCES CODING FOR DYSTROPHIN MINIGEN AND METHOD FOR USE THEREOF

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Publication number
ATE437944T1
ATE437944T1 AT01934920T AT01934920T ATE437944T1 AT E437944 T1 ATE437944 T1 AT E437944T1 AT 01934920 T AT01934920 T AT 01934920T AT 01934920 T AT01934920 T AT 01934920T AT E437944 T1 ATE437944 T1 AT E437944T1
Authority
AT
Austria
Prior art keywords
dystrophin
present
provides
minigen
dna sequences
Prior art date
Application number
AT01934920T
Other languages
German (de)
Inventor
Xiao Xiao
Original Assignee
Asklepios Biopharmaceutical In
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Asklepios Biopharmaceutical In filed Critical Asklepios Biopharmaceutical In
Application granted granted Critical
Publication of ATE437944T1 publication Critical patent/ATE437944T1/en

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)

Abstract

The present invention provides a series of novel dystrophin minigenes that retain the essential biological functions. The expression of the dystrophin minigenes may be controlled by a regulatory element along with a small polyadenylation signal. The entire gene expression cassettes may be readily packaged into a viral vector, preferably an AAV vector. The present invention further defines the minimal functional domains of dystrophin and provides ways to optimize and create new versions of dystrophin minigenes. Finally, the present invention provides a method of treatment for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).
AT01934920T 2000-04-28 2001-04-27 DNA SEQUENCES CODING FOR DYSTROPHIN MINIGEN AND METHOD FOR USE THEREOF ATE437944T1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US20077700P 2000-04-28 2000-04-28
PCT/US2001/013677 WO2001083695A2 (en) 2000-04-28 2001-04-27 Dna sequences encoding dystrophin minigenes and methods of use thereof

Publications (1)

Publication Number Publication Date
ATE437944T1 true ATE437944T1 (en) 2009-08-15

Family

ID=22743139

Family Applications (1)

Application Number Title Priority Date Filing Date
AT01934920T ATE437944T1 (en) 2000-04-28 2001-04-27 DNA SEQUENCES CODING FOR DYSTROPHIN MINIGEN AND METHOD FOR USE THEREOF

Country Status (9)

Country Link
US (2) US7001761B2 (en)
EP (1) EP1287125B1 (en)
CN (1) CN100422320C (en)
AT (1) ATE437944T1 (en)
AU (1) AU2001261063A1 (en)
CA (1) CA2407309C (en)
DE (1) DE60139394D1 (en)
ES (1) ES2330615T3 (en)
WO (1) WO2001083695A2 (en)

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USRE47769E1 (en) 2004-06-28 2019-12-17 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2007123391A1 (en) 2006-04-20 2007-11-01 Academisch Ziekenhuis Leiden Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly expressed gene.
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
US20100184833A1 (en) 2006-08-11 2010-07-22 Prosenta Technologies B.V. Methods and means for treating dna repeat instability associated genetic disorders
US7863017B2 (en) 2006-12-01 2011-01-04 Wisconsin Alumni Research Foundation TAT-utrophin as a protein therapy for dystrophinopathies
US7892824B2 (en) * 2007-01-18 2011-02-22 University Of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma
US20080221027A1 (en) * 2007-03-09 2008-09-11 The Regents Of The University Of California Composition and Methods for the Treatment of Duchene Muscular Dystrophy
CA2942716C (en) 2007-07-12 2019-07-09 Biomarin Technologies B.V. Molecules for targeting compounds to various selected organs or tissues
AU2008317566B2 (en) 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
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EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
KR20190079702A (en) 2008-10-24 2019-07-05 사렙타 쎄러퓨틱스 인코퍼레이티드 Multiple exon skipping compositions for dmd
ES2593836T3 (en) 2009-04-24 2016-12-13 Biomarin Technologies B.V. Oligonucleotide comprising an inosine to treat DMD
DK2499249T3 (en) 2009-11-12 2018-12-03 Univ Western Australia ANTISENCE MOLECULES AND PROCEDURES FOR TREATING PATHOLOGIES
US20110197290A1 (en) 2010-02-11 2011-08-11 Fahrenkrug Scott C Methods and materials for producing transgenic artiodactyls
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AU2013212758A1 (en) 2012-01-27 2014-08-14 Biomarin Technologies B.V. RNA modulating oligonucleotides with improved characteristics for the treatment of Duchenne and Becker muscular dystrophy
WO2014081855A1 (en) * 2012-11-20 2014-05-30 Universite De Montreal Methods and compositions for muscular dystrophies
CN117844807A (en) 2013-03-14 2024-04-09 萨勒普塔医疗公司 Exon skipping compositions for the treatment of muscular dystrophy
MX2015013117A (en) 2013-03-15 2016-07-14 Sarepta Therapeutics Inc Improved compositions for treating muscular dystrophy.
GB201507842D0 (en) * 2015-05-07 2015-06-17 New Royal Holloway & Bedford Production of large-sized microdystrophins in an AAV-based vector configuration
MA45477A (en) 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital ADENOASSOCIATED VIRUS VECTOR VECTOR MICROARN-29 AND MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY
CA2971303A1 (en) * 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
JP7213238B2 (en) 2017-10-18 2023-01-26 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル Adeno-associated viral vector delivery of muscle-specific microdystrophins to treat muscular dystrophy
EP3780952A4 (en) * 2018-04-16 2022-03-09 The Trustees of the University of Pennsylvania Compositions and methods for treating duchenne muscular dystrophy
MX2021005720A (en) * 2018-11-16 2021-07-21 Astellas Pharma Inc Method for treating muscular dystrophy by targeting utrophin gene.
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EP4054618A1 (en) 2019-11-06 2022-09-14 Association Institut de Myologie Combined therapy for muscular diseases
AR121954A1 (en) 2020-04-29 2022-07-27 Bristol Myers Squibb Co MINIATURIZED DYSTROPHINS WITH SPECTRIN FUSION DOMAINS AND USES THEREOF
CA3200401A1 (en) 2020-11-03 2022-05-12 Pfizer Inc. Methods for purification of aav vectors by anion exchange chromatography
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RU2767335C1 (en) * 2021-03-02 2022-03-17 Общество с ограниченной ответственностью «Марлин Биотех» Chimeric proteins based on human utrophin and dystrophin and use thereof for treating duchenne muscular dystrophy
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Also Published As

Publication number Publication date
US7001761B2 (en) 2006-02-21
WO2001083695A3 (en) 2002-03-07
EP1287125A2 (en) 2003-03-05
EP1287125A4 (en) 2005-04-27
EP1287125B1 (en) 2009-07-29
WO2001083695A2 (en) 2001-11-08
CA2407309C (en) 2011-08-02
US20060073586A1 (en) 2006-04-06
US7510867B2 (en) 2009-03-31
CN1439051A (en) 2003-08-27
WO2001083695A9 (en) 2002-12-12
CN100422320C (en) 2008-10-01
AU2001261063A1 (en) 2001-11-12
US20030171312A1 (en) 2003-09-11
ES2330615T3 (en) 2009-12-14
DE60139394D1 (en) 2009-09-10
CA2407309A1 (en) 2001-11-08

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